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Early Safety and Efficacy Data in Cynata’s Phase 1 Trial of CYP-001 in GvHD

Encouraging Early Safety and Efficacy Data in Cynata’s Phase
1 Trial of CYP-001 in GvHD; DSMB Recommendation to Initiate Enrolment
of Second Patient Cohort

Australian stem cell and regenerative medicine company, Cynata
Therapeutics Limited (ASX: CYP) announced that the independent
Data Safety Monitoring Board (DSMB) has recommended that Cynata’s clinical trial
of its lead Cymerus™ mesenchymal stem cell (MSC) product CYP-001 should
progress to the next stage as planned.

Key Highlights:

• All eight participants in Cohort A (lower dose cohort)
have demonstrated at least a Partial

Response (defined as an improvement in the severity of GvHD
by at least one grade compared

to baseline)

• No treatment-related serious adverse events or safety
concerns have been identified

• Patient enrolment in Cohort B (higher dose cohort) now
open at seven trial sites in the U.K.

and Australia

Cynata’s clinical trial, which is the first clinical trial
in which patients have been treated with an allogeneic, induced pluripotent stem cell (iPSC)-derived
therapeutic MSC product, consists of a planned total of 16 patients with steroid-resistant acute
graft-versus-host disease (GvHD). The recommendation to progress to the next stage (Cohort B) followed an
independent review by the DSMB of the eight participants in Cohort A. Recruitment for Cohort A commenced
in May 2017, and there are currently seven trial sites active and ready to enrol participants
into Cohort B.

Steroid-resistant GvHD patients today have a dismal
prognosis, where mortality rates are very high. At this time, seven of the eight participants in Cohort A are
alive. One participant died after developing pneumonia, which is a common finding in recipients of bone
marrow transplants and similar procedures.1 This death was not considered to be
treatment-related. Participants enrolled in Cohort A of the dose-escalation
trial received a dose of CYP-001 that was anticipated to be at the lower end of the effective dose
range (one million cells per kilogram of bodyweight, up to a maximum of 100 million cells per
infusion). In Cohort B, a further eight participants will receive two infusions of CYP-001 at a dose of two
million cells per kilogram of bodyweight, up to a maximum of 200 million cells per infusion.

Dr Ross Macdonald, CEO of Cynata Therapeutics, said, “We are
thrilled to report this encouraging early review of the Phase 1 trial of CYP-001, which marks the
first time that patients have been treated with an allogeneic, induced pluripotent stem cell-derived
therapeutic MSC product. The improvement in GvHD grade observed in 100% of these gravely ill people is
very promising, especially given the low dose administered in Cohort A. The positive DSMB recommendation
is an important milestone that enables us to begin enrolment in Cohort B, and advance toward our
goal of completing the trial later this year. A successful outcome will support the application of CYP-001
in many medically and commercially significant targets where therapeutic MSCs have shown
promising results.”

Next Steps:

Patient enrolment into Cohort B is now open at seven active
sites across the U.K. and Australia. Cynata looks forward to providing further updates to the market as
the study progresses.